The development of new drugs is a long and expensive process, taking many years and huge amounts of funding to bring novel and better therapeutics to treat illnesses in people. Drug repurposing offers a means of rapidly getting new therapies to patients by clinically testing already available medicines in diseases that they were not originally approved for.

When a drug is first tested in people, much of the analysis focuses on the long-term safety and tolerability of the treatment. A drug that is already approved by clinicians to treat a particular medical condition will already have a well-documented history of safe clinical use. This knowledge allows investigators to fast-track a large proportion of the clinical testing process. If preclinical efficacy data is also supportive, the repurposing of that drug for another disease can occur relatively quickly.

Cure Parkinson’s has been using the drug repurposing approach for 10 years in our International Linked Clinical Trials (iLCT) programme, and recently there have been a number of preclinical studies providing supportive evidence for additional drugs that could be repurposed for Parkinson’s.

Recent research reports have highlighted new data for the repurposing of clinically available drugs for Parkinson’s. A new report, from the lab of iLCT committee member Professor Dimitri Krainc in Chicago, has found that an anti-psychotic medication, quetiapine, can activate and increase levels of a Parkinson’s-associated enzyme called GCase. This enzyme is reduced in Parkinson’s, and Professor Krainc and his team are hoping that quetiapine can be repurposed for people with Parkinson’s to help biologically improve GCase activity.

Another report has identified the heart-failure drug, bumetanide, as a potential agent for treating neurodegenerative conditions. People who carry a genetic variation called APOE4 make up about 1/4 of the population and have a higher risk of developing Alzheimer’s and other cognitive issues associated with aging. APOE4 has also been found to influence the progression of Parkinson’s. Repurposing bumetanide to treat carriers of this genetic variation could have important implications for Parkinson’s, as well as major benefits for society as a whole.

As Cure Parkinson’s continues to champion the repurposing of therapies for Parkinson’s, it is encouraging and exciting to see additional drugs coming forward as candidates for this programme of research. It is hoped these will lead to further treatments and quality of life options for the Parkinson’s community.

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